Tagged with Rare Diseases

Europe 125 medicines for rare diseases were approved by the EMA between 2004 and 2014, of which 71 were granted orphan drug status. This chart shows the availability of 112 of these drugs across European countries. The largest number of medicines for rare diseases were in continuous use in Germany and…

Turkey Levent Canyurt, CEO and founder of BIEM Ilac, shares how he transformed the company from a domestic pharmaceutical distributor into an international player, with an innovative portfolio for the treatment of rare diseases. He also highlights the importance of greater collaboration between stakeholders in the Turkish biotech industry and gives…

Turkey Abidin Gulmus, founder and chairman of Gen Ilaç, describes the company’s impressive journey to become Turkey’s principle orphan drug distributor and shares his vision for leveraging the company’s domestic expertise to expand their reach into foreign markets.   Gen Ilaç is one of the fastest growing Turkish success stories although…

Malaysia Sea cucumbers – odd-looking marine animals with leathery skins and elongated bodies – can be found on ocean floors worldwide. Although more famous as a culinary delicacy in East and Southeast Asia, one Malaysian company – Healwell – is using these primitive creatures to save lives.   Dr Hassan Yaakob,…

Korea Korean Pharma company, Hanmi has won orphan drug status this week from the US Food and Drug Administration (FDA) for HM43239, a drug for the treatment of acute myeloid leukaemia (AML).   The endorsement of FDA’s Orphan Drug Designation (ODD) — invented to smooth the development of therapies for life-threatening…

Innovation Patient groups oftentimes serve as the authority on a specific disease when it comes to advancing research.   “Without patient perspective, there is no good medical product development,” Rachel Sherman, the FDA’s principal deputy commissioner proclaimed during her keynote at last week’s NORD’s Rare Disease & Orphan Products Breakthrough Summit.…

Mexico David Peña Castillo is the President and founder of The Mexican Federation of Rare Diseases (FEMEXER). Here he discusses what makes a patients association. When we find a way to communicate the complexities of rare diseases simply, we can avoid isolating anyone and the potential to uncover important information is…

USA After her son was diagnosed with SYNGAP1 – an extremely rare gene mutation – Monica founded Bridge the Gap – SYNGAP Education and Research Foundation to support families of those suffering from the illness and to accelerate the path to better therapies. Here she discusses the importance of community for tackling rare…

France Genethon, a laboratory created by the patient organization AFM-Telethon, is one of the world’s leaders in the development of biotherapies for rare diseases. In this interview Genethon’s CEO Frédéric Revah explains its role in the discovery of innovative therapies since the 1990s and the recent launch of YposKesi, its innovative…

Turkey Two months into the role, Umut Meriç, general manager for Chiesi Turkey, discusses the affiliate’s rapid progression and his plans to ensure Turkish patients benefit from the global company’s latest therapies in the respiratory and rare diseases areas. Meriç underlines the importance of motivating and developing a productive, dynamic and…

UK Offering genomic sequencing and analysis, Congenica operates in the exciting world of genomic diagnosis, with a particular focus on rare diseases. Its CEO David Atkins describes the potential of Congenica’s platform and talks commercialisation. Can you please start by introducing Congenica to our international readers? “As well as disease diagnosis,…

UK Sean Richardson discusses Alexion’s focus on rare and ultra-rare diseases, the level of support for these therapies from the UK government, awareness raising, diagnostics, and his future priorities.     Let’s please start this interview by introducing the UK affiliate of Alexion to our audience. How would you describe the…